At BOLDAPPROVALS, we support clients in preparing for FDA Advisory Committee meetings, developing communication strategies for regulatory submissions, and preparing for formal interactions with FDA. Our approach to communication strategy, content development, and delivery can help sponsors make the most of these opportunities.

Learn how we can prepare your team for a CTGT AdComm at boldapprovals.com, info@boldapprovals.com, or call us at +1 (646) 930-0453.

It’s challenging to prepare for a meeting of the Advisory Committee with the widest range of expertise, far greater than any other. It’s crucial to understand the diversity of expertise on the standing committee, plus that of the likely temporary voting member. To succeed, sponsors need to combine persuasive data with finely tuned education.

• CTGT brings the broadest range of expertise of any Advisory Committee
• Temporary voting members may be deeply specialized by indication, while many standing members are not, especially in rare diseases
• Success requires both education and persuasive data to keep the Committee engaged and aligned
• An audience-focused communication strategy is essential to align the Committee and enable confident decision-making

CTGT has the most diverse expertise of any FDA Advisory Committee – and, as a result, is one of the easiest to misjudge. Sponsors who fail to align this heterogeneous audience risk losing Committee support, even when the data are strong. Many of its members are actively engaged in the same sort of research that sponsors are presenting. They are variously involved in tissue and stem cell transplantation, xenotransplantation, gene therapy, virology, hematology, oncology, neurology, cardiology, and radiology. This breadth of expertise enables CTGT to evaluate a wide range of products across therapeutic areas and treatment modalities, but it also creates a uniquely complex communication environment for sponsors.

FDA typically supplements the standing Committee with temporary voting members with areas of expertise specific to the issues at hand for a given product. As a result, the spectrum of product-specific expertise can be exceptionally broad, ranging from subject matter experts with in-depth knowledge to those working in the same therapeutic area but not the specific indication, as well as members whose expertise lies in entirely different fields. While this versatility is an advantage for the range of products the Committee can review, it also creates challenges for sponsors with complex topics or rare-disease indications that are familiar to only a few temporary voting members but unfamiliar to most of the standing Committee.

Fortunately, members of this Committee are used to evaluating questions beyond their primary area of expertise. Nonetheless, it is critical for sponsors to bring the entire Committee along throughout their presentations and Q&A. This requires achieving a level of clarity that reaches across the spectrum of expertise of the Committee while maintaining credibility with members who have deep knowledge of the product class and therapeutic area. With rare diseases and/or novel therapeutic approaches, such as first-in-class or first-in-indication treatments, sponsors must actively educate the Committee while presenting persuasive data. This can be particularly challenging when submissions include a novel endpoint that require explanation of clinical relevance and the meaningfulness of observed changes.

In preparing for a CTGT meeting, sponsors must therefore both educate and persuade. This can include education on disease state and pathophysiology, treatment and mechanism of action, endpoint selection and clinical meaningfulness, and associated risks. These needs can be greatest with novel therapies for rare diseases, particularly first-in-class and/or first-in-indication treatments. At the same time, sponsors must recognize that some Committee members may already be well-versed in the disease and therapeutic approach.

By combining education with persuasive data, sponsors can align these diverse members on a clear path to approval

For example, a client of ours had a gene therapy for a rare genetic disorder that represented the first disease-specific treatment. Although their ex vivo approach using an established vector was familiar to the Committee, the disease state, disease course, and etiology were not—nor was the primary endpoint. In addition, as is often the case with gene therapies for genetic diseases, the timing of treatment was highly predictive of treatment outcome such that the earlier treatment is initiated, the better the outcome. To address this, it was crucial for the sponsor to first educate the Committee on the natural history of the disease, enabling members to understand what progression looks like at different stages of childhood development. This foundation allowed the Committee to interpret treatment effects in context and appreciate differences in outcomes based on timing of diagnosis and treatment initiation.

This example illustrates the importance of developing a communication strategy based on the specific audience. For CTGT, that means understanding the diversity of expertise, addressing complexity directly rather than oversimplifying, and tailoring communication to both educate and persuade Committee members with data-driven messages tuned to their varying areas of expertise. By following this approach, our client was able to effectively balance education and persuasion, aligning a Committee with highly divergent expertise.

With the right support, a sponsor team can deliver compelling content that focuses on what matters most to the Committee, supported by a clear and persuasive narrative of the disease and study results.